18200950. COMPOUNDS FOR TAU PROTEIN DEGRADATION simplified abstract (DANA-FARBER CANCER INSTITUTE, INC.)

COMPOUNDS FOR TAU PROTEIN DEGRADATION

Organization Name

DANA-FARBER CANCER INSTITUTE, INC.

Inventor(s)

Nathanael S. Gray of Boston MA (US)

Stephen J. Haggarty of Gloucester MA (US)

Quan Cai of Shanghai (CN)

Tinghu Zhang of Brookline MA (US)

Maria Catarina Telo Baptista Lima Da Silva of Revere MA (US)

Fleur M. Ferguson of Cambridge MA (US)

COMPOUNDS FOR TAU PROTEIN DEGRADATION - A simplified explanation of the abstract

This abstract first appeared for US patent application 18200950 titled 'COMPOUNDS FOR TAU PROTEIN DEGRADATION

Simplified Explanation

The patent application describes bifunctional compounds that bind tau protein and promote targeted ubiquitination for its degradation. These compounds can recruit an E3 ubiquitin ligase to mark tau protein for proteasomal degradation.

• Bifunctional compounds bind tau protein and promote its degradation by recruiting an E3 ubiquitin ligase.
• E3 ubiquitin ligase marks tau protein for proteasomal degradation.

Potential Applications

The technology could be used in:

• Developing new treatments for neurodegenerative diseases.
• Improving methods for detecting and diagnosing neurological disorders.

Problems Solved

This technology addresses:

• Pathological aggregation of tau protein in the central nervous system.
• Lack of effective treatments for tauopathies and other neurodegenerative diseases.

Benefits

The benefits of this technology include:

• Targeted degradation of tau protein implicated in neurodegenerative diseases.
• Potential for more accurate diagnosis and monitoring of neurological disorders.

Potential Commercial Applications

A potential commercial application of this technology could be:

• Developing novel pharmaceuticals for the treatment of neurodegenerative diseases.

Possible Prior Art

One possible prior art could be:

• Previous research on tau protein aggregation and degradation pathways in neurodegenerative diseases.

Unanswered Questions

How scalable is the production of these bifunctional compounds for widespread use in clinical settings?

The scalability of production for clinical use is not addressed in the patent application. Further research and development may be needed to optimize production processes for large-scale applications.

What are the potential side effects or limitations of using compounds that promote tau protein degradation in vivo?

The potential side effects or limitations of using these compounds in vivo are not discussed in the patent application. Additional studies and clinical trials would be necessary to evaluate the safety and efficacy of these compounds for human use.

Original Abstract Submitted

Provided herein are bifunctional compounds that bind tau protein and/or promote targeted ubiquitination for the degradation of tau protein. In particular, provided are compounds that can bind tau protein, a protein whose aggregation is implicated in a variety of neurodegenerative disease (e.g., tauopathies), and can promote its degradation by recruiting an E3 ubiquitin ligase (e.g., Cereblon), which can ubiquitinate tau protein, marking it for proteasomal degradation. Also provided are radiolabeled forms of the bifunctional compounds, pharmaceutical compositions comprising the bifunctional compounds, methods of detecting and/or diagnosing neurological disorders, methods of detecting and/or diagnosing pathological aggregation of tau protein (e.g., in the central nervous system), methods of treating and/or preventing neurological disorders, and methods of promoting the degradation of tau protein by E3 ubiquitin ligase activity in a subject by administering a compound or composition described herein.